Sevion Therapeutics, Inc. and Eloxx Pharmaceuticals Ltd., a clinical-stage biopharmaceutical company developing novel translational read-through inducing drugs (TRIDs) for genetic diseases caused by nonsense mutations, recently announced dosing of the first subject in its multiple ascending dose (MAD) Phase 1b clinical trial with its lead candidate, ELX-02. The double-blind, placebo-controlled, dose-escalating trial will enroll 36 subjects across four dose levels, and is designed to evaluate the safety, tolerability, and pharmacokinetics of ELX-02 in healthy volunteers (see www.clinicaltrials.gov, NCT03309605). The trial is being run in Belgium, and data are expected at the end of the fourth quarter 2018.

“We are very pleased to continue our clinical development program for ELX-02 and are excited about its potential to treat many genetic diseases caused by nonsense mutations,” said Pedro Huertas, MD, PhD, Chief Medical Officer of Eloxx Pharmaceuticals. “We look forward to continue achieving our key clinical milestones.”
ELX-02 is a fifth-generation designer aminoglycoside optimized by medicinal chemistry approaches to act as a translational read-through inducing drug (TRID), enabling the synthesis of full-length proteins. ELX-02 has the potential to treat many rare genetic diseases with high prevalence of nonsense mutations, such as cystic fibrosis, cystinosis, mucopolysaccharidosis Type I (MPS I), Rett Syndrome, and Duchenne muscular dystrophy (DMD).

Eloxx Pharmaceuticals is a clinical-stage biopharmaceutical company developing novel small molecule medicines to treat many rare and ultra-rare genetic diseases caused by nonsense mutations. Nonsense mutations are a class of gene defect that result in premature termination of protein synthesis. As a consequence, patients with a genetic disease caused by nonsense mutations have absent or truncated nonfunctional proteins, accounting for some of the most severe phenotypes in these genetic diseases. Eloxx’s lead product candidate, ELX-02, is an optimized aminoglycoside designed to restore full-length functional proteins. Eloxx Pharmaceuticals was founded in 2013 and maintains offices in Boston, MA, and Rehovot, Israel.

NeuroproteXeon, Inc. announced the publication of a second finding from a randomized, controlled Phase II trial of inhaled xenon and oxygen, combined with hypothermia, for out-of-hospital cardiac arrest patients. The study, which appeared in a recent issue of the Journal of the American College of Cardiology (JACC Vol. 70, No. 21, November 28, 2017: 2652-60) found, "among comatose survivors of OHCA, in comparison with hypothermia alone, inhaled xenon along with hypothermia suggests less severe myocardial injury as demonstrated by the significantly reduced level of troponin-T" P= 0.01.

NeuroproteXeon is preparing to start its pivotal, 30-center, Phase III trial in the second quarter of 2018. The Company has an agreed Special Protocol Assessment (SPA) with the US FDA and a Scientific Advisory Working Party Agreement (SAWP) with the European Medicines Agency (EMA). Both FDA and EMA have provided the Company with orphan designation on this application with related market exclusivity (7 and 12 years, respectively).

NeuroproteXeon, Inc. is a clinical-stage pharmaceutical/device company developing xenon to improve functional capabilities and survival in patients with Post-Cardiac Arrest Syndrome (PCAS). The Company has a license agreement with Mallinckrodt LLC for the sale and distribution of xenon gas for inhalation in North America, Australia and Japan upon approval of the drug in those markets.